ZILGANERSEN

U.S. Department of Health & Human Services Divider Arrow

National Institutes of Health Divider Arrow

NCATS

Approval Year

Substance Class	Nucleic Acid Created by `admin` on `Wed Apr 02 08:59:17 GMT 2025` Edited by `admin` on `Wed Apr 02 08:59:17 GMT 2025`
Nucleic Acid Type	MRNA
Sequence Origin	SYNTHETIC
Sequence Type	COMPLETE
Record UNII	AXQ9493NT2
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

ION-1166998 FREE ACID

Preferred Name

English

ZILGANERSEN

Official Name

English

DNA, D((2'-O-(2-METHOXYETHYL))M5RC-SP-(2'-O-(2-METHOXYETHYL))RA-(2'-O-(2-METHOXYETHYL))RG-(2'-O-(2-METHOXYETHYL))M5RU-(2'-O-(2-METHOXYETHYL))RA-(2'-O-(2-METHOXYETHYL))M5RU-T-SP-A-SP-M5C-SP-M5C-SP-T-SP-M5C-SP-T-SP-A-SP-M5C-SP-T-SP-(2'-O-(2-METHOXYETHYL))R

Common Name

English

zilganersen [INN]

Common Name

English

ZILGANERSEN [USAN]

Common Name

English

Code System Code Type Description

INN

11915

Created by admin on Wed Apr 02 08:59:17 GMT 2025 , Edited by admin on Wed Apr 02 08:59:17 GMT 2025

PRIMARY

USAN

HI-42

Created by admin on Wed Apr 02 08:59:17 GMT 2025 , Edited by admin on Wed Apr 02 08:59:17 GMT 2025

PRIMARY

SMS_ID

300000039921

Created by admin on Wed Apr 02 08:59:17 GMT 2025 , Edited by admin on Wed Apr 02 08:59:17 GMT 2025

PRIMARY

FDA UNII

AXQ9493NT2

Created by admin on Wed Apr 02 08:59:17 GMT 2025 , Edited by admin on Wed Apr 02 08:59:17 GMT 2025

PRIMARY

CAS

2305355-56-8

Created by admin on Wed Apr 02 08:59:17 GMT 2025 , Edited by admin on Wed Apr 02 08:59:17 GMT 2025

PRIMARY

NCI_THESAURUS

C186394

Created by admin on Wed Apr 02 08:59:17 GMT 2025 , Edited by admin on Wed Apr 02 08:59:17 GMT 2025

PRIMARY

Sugar	Site Range	Site Count
Deoxyribose (DNA)	1_7-1_16	10 / 20
2'-O-Methoxyethyl ribose	1_1-1_6;1_17-1_20	10 / 20

Linkage	Site Range	Site Count
Phosphate	1_3-1_7;1_18	6 / 19
Phosporothioate	1_2;1_8-1_17;1_19-1_20	13 / 19

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Amount:


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GLIAL FIBRILLARY ACIDIC PROTEIN

EXPRESSED PROTEIN->VECTOR

Amount:

Related Record Type Details


					AXQ9493NT2

ACTIVE MOIETY

Comments:

For the treatment of Alexander disease (AxD)

Amount:

Structural Modifications

Modification Type	Location Site	Location Type	Residue Modified	Extent	Fragment Name	Fragment Approval
NUCLEOSIDE BASE SUBSTITUTION	[1_4] [1_6]	SITE_SPECIFIC			THYMINE	QR26YLT7LT
NUCLEOSIDE BASE SUBSTITUTION	[1_1] [1_9] [1_10] [1_12] [1_15] [1_20]	SITE_SPECIFIC			5-METHYLCYTOSINE	6R795CQT4H